Sarepta Therapeutics Executes Licensing Agreement for Gene Therapy Program from Nationwide Children’s Hospital to Treat Limb-Girdle Muscular Dystrophy Type 2A
August 04 2021 - 8:30AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today announced that upon
completion of a number of preclinical and safety studies, it had
executed an exclusive license agreement for an investigational gene
therapy candidate, calpain 3 (CAPN-3), to treat Limb-girdle
muscular dystrophy type 2A (LGMD2A), developed by the Abigail
Wexner Research Institute at Nationwide Children’s Hospital
(Nationwide Children’s).
LGMDs represent a group of distinct genetic
neuromuscular diseases with a generally common set of symptoms,
including progressive, debilitating weakness and wasting that
begins in muscles around the hips and shoulders before progressing
to muscles in the arms and legs. Many LGMD sub-types are
significantly life-limiting and often life-ending diseases. Also
known as calpainopathy, LGMD2A is caused by mutations in the CAPN-3
gene and is the most common type of LGMD, accounting for almost a
third of cases.
“Treatment plans for LGMD2A are currently
limited to physical therapy, assistive devices and surgery for
complications. We’re excited about the opportunity to transform
patient care for this significantly life-limiting disease by
advancing the CAPN-3 program following extensive pre-clinical work
by the team at Nationwide Children’s. Preclinical research
conducted to date has provided early proof of concept for CAPN-3 in
LGMD2A and supports further advancement,” said Louise
Rodino-Klapac, Sarepta’s executive vice president and chief
scientific officer. “We intend to build off the knowledge we have
gained from our lead investigational gene transfer programs for
Duchenne muscular dystrophy and LGMD2E, as the CAPN-3 program also
uses the AAVrh74 vector to address another well-characterized
genetic disease. Sarepta’s commitment and research investment in
LGMD is unparalleled and we continue to work towards advancing all
of our LGMD programs as quickly as possible.”
Like SRP-9001, Sarepta’s lead investigational
gene transfer therapy for Duchenne muscular dystrophy, and the
Company’s five other LGMD programs, the LGMD2A program uses the
AAVrh74 vector, designed to systematically and robustly deliver
treatment to skeletal muscle, including the diaphragm, making it an
ideal candidate to treat muscle disease.
The preclinical work on the CAPN-3 program in
LGMD2A has been led by Zarife Sahenk, M.D., Ph.D., attending
neurologist at Nationwide Children’s, Director of Clinical and
Experimental Neuromuscular Pathology at The Research Institute at
Nationwide Children’s and Professor of Pediatrics, Pathology and
Neurology at The Ohio State University College of Medicine.
About Limb-girdle Muscular
DystrophyLimb-girdle muscular dystrophies are genetic
diseases that cause progressive, debilitating weakness and wasting
that begins in muscles around the hips and shoulders before
progressing to muscles in the arms and legs. Sarepta’s six LGMD
gene therapy programs in development include LGMD2E, LGMD2D,
LGMD2C, LGMD2B, LGMD2L and LGMD2A, which together represent more
than 70 percent of known LGMD cases.
About Sarepta
TherapeuticsSarepta is on an urgent mission: engineer
precision genetic medicine for rare diseases that devastate lives
and cut futures short. We hold leadership positions in Duchenne
muscular dystrophy (DMD) and limb-girdle muscular dystrophies
(LGMDs), and we currently have more than 40 programs in various
stages of development. Our vast pipeline is driven by our
multi-platform Precision Genetic Medicine Engine in gene therapy,
RNA and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Internet
Posting of InformationWe routinely post information that
may be important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Forward-Looking StatementsThis press release
contains "forward-looking statements." Any statements contained in
this press release that are not statements of historical fact may
be deemed to be forward-looking statements. Words such as
"believes," "anticipates," "plans," "expects," "will," "intends,"
"potential," "possible" and similar expressions are intended to
identify forward-looking statements. These forward-looking
statements include statements regarding the potential benefits of
the licensing agreement; the design of the AAVrh74 vector to
systematically and robustly deliver treatment to skeletal muscle,
including the diaphragm, making it an ideal candidate to treat
muscle disease; the potential of our portfolio of investigational
gene therapies for LGMD to address six LGMD subtypes, which
together represent more than 70% of all known LGMDs; and our plan
to continue to advance all of our LGMD programs as quickly as
possible.
These forward-looking
statements involve risks and uncertainties, many of which are
beyond our control. Known risk factors include, among others: the
expected benefits and opportunities related to the licensing
agreement may not be realized or may take longer to realize than
expected due to challenges and uncertainties inherent in product
research and development. In particular, activities under the
license may not result in any viable treatments suitable for
commercialization due to a variety of reasons, including any
inability of the parties to perform their commitments and
obligations under the agreement; success in preclinical trials does
not ensure that later clinical trials will be successful; Sarepta
may not be able to execute on its business plans and goals,
including meeting its expected or planned regulatory milestones and
timelines, clinical development plans, and bringing its product
candidates to market, due to a variety of reasons, many of which
may be outside of Sarepta’s control, including possible limitations
of company financial and other resources, manufacturing limitations
that may not be anticipated or resolved for in a timely manner,
regulatory, court or agency decisions, such as decisions by the
United States Patent and Trademark Office with respect to patents
that cover Sarepta’s product candidates and the COVID-19 pandemic;
even if Sarepta’s programs result in new commercialized products,
Sarepta may not achieve the expected revenues from the sale of such
products; if the actual number of patients living with LGMD2A
is smaller than estimated, Sarepta’s revenue and ability to achieve
profitability may be adversely affected; and those risks identified
under the heading “Risk Factors” in Sarepta’s most recent Annual
Report on Form 10-K for the year ended December 31, 2020, and most
recent Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) as well as other SEC filings made by the
Company which you are encouraged to review.
Any of the foregoing
risks could materially and adversely affect the Company’s business,
results of operations and the trading price of Sarepta’s common
stock. For a detailed description of risks and uncertainties
Sarepta faces, you are encouraged to review the SEC filings made by
Sarepta. We caution investors not to place considerable reliance on
the forward-looking statements contained in this press release.
Sarepta does not undertake any obligation to publicly update its
forward-looking statements based on events or circumstances after
the date hereof.
Source: Sarepta Therapeutics, Inc.
Investor Contact: Ian Estepan,
617-274-4052iestepan@sarepta.com
Media Contact: Tracy Sorrentino,
617-301-8566tsorrentino@sarepta.com
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